Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid

This study has been completed.

Sponsor:

Assistance Publique Hopitaux De Marseille

ClinicalTrials.gov Identifier:

NCT00731016

First Posted: August 8, 2008

Last Update Posted: July 8, 2013

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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We suggest treating the Hutchinson-Gilford Progeria Syndrome by two molecules (zoledronic acid and pravastatin).The therapeutic approach which we propose has for objectives to reduce, to prevent or to delay the gravest infringements of the disease, to prolong the life of the children, and in a more general way, aim at improving their living conditions.

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Ages Eligible for Study:

3 Years and older (Child, Adult, Senior)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Molecularly characterised patients with a known mutation of their LMNA gene leading to the production of a farnesylated prelamin A, whether truncated or not

Patients must be able to travel and consult in Marseille, France for necessary explorations planned at the inclusion step, then following the protocol flow

chart for zoledronic acid injections and follow-up visits

Patient older than 3 years

Patients affiliated or beneficiary of a legal medical insurance

Adult patients certifying they have been properly informed about the protocol, and they signed a written consent form. Children and/or disabled patients whose parents/legal tutor have been informed and have signed a written consent form

Exclusion Criteria:

Known hypersensitivity to pravastatin or zoledronic acid

Seric transaminase levels higher than 3 times of normal value

CPK level higher than 5 times of normal value

Creatininemia higher than 0.5mg/dl or 44mM, or creatinin clearance lower than 70ml/min/1.73m3